Mayuri Saxena first knew something was wrong when she started tripping at work and struggling to get out of her seat during her commute on the New York subway. In just three months, the 32-year-old got into a wheelchair, but she finally learned what was wrong: she had ALS.
Faced with a life expectancy of two to five years due to amyotrophic lateral sclerosis, a degenerative neuromuscular disease that widely affects voluntary muscles, including the vocal cords, Saxena and her family turned to experimental drug trials as their only chance to treat the disease. One by one, she was rejected either because of her wheelchair use or because she was too far advanced in her illness.
“It’s honestly that our hope started to fade,” said Mayak Saxena, Mayuri’s brother. “To know that a treatment like this exists on this planet and on the other hand to see my sister suffer, die and suffer. It is such an injustice that needs to be addressed on so many fronts.
A new bill submitted to Congress may soon offer hope to Saxena and others with ALS who have not been able to do clinical trials. Bill HR8662 and its Senate companion aim to give ALS patients early and expanded access to medical treatments in their stage 3 clinical phase. This would speed up access to potentially life-saving treatments for those with limited time. It is estimated that 30,000 people in the United States are infected with ALS and 6,000 die from it each year. There is no current treatment for the disease.
Proponents say that despite the risks associated with using an unfinished drug, the law would give ALS patients a chance to live where there currently is none. And they hope to get the legislation passed before the current Congress shuts down at the end of the year, warning that if the bill is pushed to the next session, more patients in need will die.
“Everyone in the ALS community says, ‘I’m going to die 100%. Without a choice, you can’t give me anything that will kill me, when I wouldn’t be dead. There is a different risk / benefit analysis for me, let me make that choice, ”said Michelle Lorenz of Everything ALS, a patient-focused non-profit organization. “We will lose 12,000 more people if we don’t [pass it this year], when we will reintroduce it in the next session.
The Ice Bucket Challenge 2014 is largely attributed to the increase in national recognition of the name of ALS, also known as Lou Gehrig’s disease. The viral social media stunt that prompted friends to challenge each other to throw buckets of ice water at their heads ultimately raised $ 115 million for one of the leading nonprofits in ALS.
But today, most diagnosed patients still have few treatment options other than tidying up their homes and implementing a palliative care plan, a reality that creates what some patients refer to as. “Defeatism of ALS”. And there is still a lot that we don’t know about the disease. For 10% of patients, including Saxena, the disease is genetic, but for others, the cause is unknown. The disease is known to disproportionately affect white males over the age of 60. Some experts believe there is a link between chemicals found on military bases and elsewhere that explains why so many veterans have been diagnosed with ALS.
Diagnosing patients with ALS is also tricky and can take years – a major limiting factor for patients who wish to participate in the handful of experimental ALS trials. The companies developing the drugs set strict standards for the ideal test subject to measure results by. Less than 10% of the ALS population qualifies for a trial.
It’s a reality that can be hard to digest for ALS patients and families who have no other choice.
“It’s infuriating, because it’s like, well, why do these trials even exist?” What’s the point of compassionate use if there isn’t even compassion they are using, ”Mayak Saxena said of her sister’s experience.
“We are already written off. So really for us all we try to do is, you know, the risk factor is zero, basically.“
This has led to a heightened push by advocates and a growing sense of anger among patients over the Federal Drug Administration’s limits on access to drugs.
“ALS has always been a disease where, due to the time scenario and the age of the people it normally strikes, there has been no time for manifestations. There was no time to get angry with this disease. And that’s changing, ”said Mike Henson, an ALS patient who is part of the online patient advocacy group No More Excuses. “Patients have had enough of this shit.”
Henson knows how important access to trial drugs is for people with ALS. Earlier this year, he was fortunate enough to participate in the stage 3 trial for the drug NurOwn and found success. During the few months he was on treatment, his illness did not worsen.
Still, it took NurOwn 10 years to develop at this point, and it is still not commercially available. Even Henson cannot access it now that the trial is over and he said he has already seen his motor functions start to decline.
When he found out he had ALS two years ago at the age of 47, Henson said he had gone to a “pretty dark place”.
“You don’t live with ALS, you die with ALS,” he says.
Among drugs, Henson said access was everything.
“We are already written off. So really for us all we try is, you know, the risk factor is basically zero.
In addition to opening logistical access to patients, the law also authorizes $ 100 million in funding to cover the cost of drugs for patients so that there are no out-of-pocket expenses. Supporters of the federal bill are doing all they can to get it enacted before this Congress ends, in less than two months. Any delay means more lives lost.
For some of the members of Congress sponsoring the bill, the passage is also personal. Rep. Jeff Fortenberry (R-NE) lost his brother-in-law to ALS last April. Senator Lisa Murkowski (R-AL) also has a step-cousin who died of the disease.
So far, there are 75 co-sponsors on the House bill and advocates are eagerly awaiting the Chairman of the House Energy and Commerce Committee, Frank Pallone (D-NJ), submits it to the committee for a full vote.
It has been difficult for some to witness the slow exploration of the bipartisan bill, especially as they see COVID-19 drugs like remdesivir being speeded up and put on the green light for use by the consumers in a revolutionary period of time.
“I think in the ALS community people are really angry, like, ‘Why do you have access to a drug and I can’t?
Advocates and lawmakers know it will be an uphill battle for the ALS bill to pass in a lame duck session emerging from a tumultuous presidential election. They have less than two months to hand it over to President Trump’s office.
“We have the logistical challenges of people who are not in their offices, you know, but despite that we still have to move forward because we have worked so hard to get to where we are now. And we’re about six weeks into the New Year. So we were really on the phone, ”said Mayak Saxena.
“But, you know, we always put our hats off on hope.”
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